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找到 38,649 条关于
“multiple sulfatase deficiency”
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去年
FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Six Gene Therapies
2年前
JCR Pharmaceuticals' JR-441 Receives Clearance for Phase I MPS IIIA Trial in Japan
2年前
FDA Extends Review Timeline for REGENXBIO's Hunter Syndrome Gene Therapy RGX-121
去年
Denali Therapeutics' Tividenofusp Alfa Receives FDA Breakthrough Therapy Designation for Hunter Syndrome
去年
GC Biopharma's Hunterase ICV Approved in Russia for Severe Hunter Syndrome
9个月前
Immusoft Receives FDA Orphan Drug Designation for Engineered B Cell Therapy ISP-002 in Hunter Syndrome
去年
JCR Pharmaceuticals Completes Enrollment in Global Phase III Trial for Blood-Brain Barrier-Penetrating Hunter Syndrome Therapy
去年
FDA Approves Kebilidi, First Gene Therapy Directly Administered to the Brain for AADC Deficiency
去年
FDA Approves Kebilidi, First Gene Therapy for AADC Deficiency
去年
FDA Approves Kebilidi, First Gene Therapy for AADC Deficiency
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